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Article

Ivacaftor in cystic fibrosis with residual function: Lung function results from an N-of-1 study.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2020

Nick Jerry A, St Clair Connie, Jones Marion C, Lan Lan, Higgins Mark

Abstract excerpt

BACKGROUND: Ivacaftor shows benefit in patients with cystic fibrosis (CF) and CFTR mutations associated with residual CF transmembrane conductance regulator (CFTR) function. Here we further assess the effect of ivacaftor in such patients using an N-of-1 study design. METHODS: Patients aged ≥12 years with CF with clinical or molecular evidence of residual CFTR function were randomized to 1 of 4 treatment sequences...

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Identifiers

PMID: 31784217
DOI: 10.1016/j.jcf.2019.09.013

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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